A Study of AAV-hAADC-2 in Subjects With Parkinson's Disease

This study has been completed.

Sponsor:

Genzyme, a Sanofi Company

ClinicalTrials.gov Identifier:

NCT00229736

First Posted: September 30, 2005

Last Update Posted: December 4, 2013

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
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The safety and tolerability of intrastriatal administration of AAV-hAADC-2 as measured by Adverse Events in subjects with mid- to late-stage Parkinson's Disease. [ Time Frame: Time of treatment through Month 60. ]

* Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ICMJE

Completed

Enrollment ICMJE

10

Completion Date

March 2013

Primary Completion Date

March 2013 (Final data collection date for primary outcome measure)

Eligibility Criteria ICMJE

Inclusion Criteria:

Documented informed consent.

Diagnosis of idiopathic Parkinson's disease (two of four cardinal features - tremor, bradykinesia, rigidity and postural instability - plus early dopaminergic responsiveness of symptoms and absence of any cardinal signs of more widespread neurological disease).

Age ≤ 75 years.

Age at diagnosis ≥ 40 years.

Duration of levodopa therapy ≥ 5 years (number of years is cumulative since diagnosis, does not need to be continuous).

Positive response to dopaminergic therapy as evidenced by a decrease in UPDRS motor scores between the defined "OFF" and "ON" states: a minimum of 8 points improvement in the UPDRS after dopaminergic therapy.

Candidate for surgical therapy for Parkinson's disease because of intractable motor fluctuations, defined as a subscore on UPDRS Part IV section B ("Clinical Fluctuations" score of 3 (minimum) to 7 (maximum) during the "ON" state, not responsive to optimal medical therapy.

MRI (magnetic resonance imaging)within the past two years and MRI does not show any conditions that are clinically significant with respect to risks for brain surgery.

Subjects must agree to use barrier contraception, until three consecutive PCR (polymerase chain reaction) samples are negative, if the subject or partner is of childbearing potential.

Must be able to comply with the requirements of the study, including the need for frequent and prolonged follow-up.

Subjects must have been on both optimal and stable medications for treatment of their Parkinson's disease for at least two months prior to being eligible for participation in the study.

Subjects must have baseline Hematology and Chemistry values within the normal laboratory ranges unless the out of range values are not clinically significant with respect to general surgery.